“A PFIC diagnosis for our children was shocking and devastating, and we quickly found out that current treatment options are inadequate,” said Kristen and Michael Busby of New York, parents of two children with PFIC. “We applaud all research in this area, and it is great news to see a clinical trial as an option for children with PFIC.”

We applaud all research in this area, and it is great news to see a clinical trial as an option for children with PFIC

Kristen and Michael Busby ​

PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease. Moderate to severe pruritus is a common and problematic clinical presentation of PFIC that can severely diminish quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all patients with PFIC require treatment before age 30. There are currently no approved pharmacological treatment options for PFIC. “We are very excited to have the first patient enrolled into the PEDFIC-1 pivotal study to determine the potential of A4250, with the goal of providing new options to patients with this debilitating disease,” said Professor Richard Thompson, King's College London and principal investigator of the study. “The results of the Phase 2 trial, where A4250 reduced serum bile acids and decreased pruritus in most patients, while showing a favourable overall tolerability profile, gives us optimism and confidence that A4250 could be an excellent medical treatment option.” The Phase 3 program includes a single randomized, double-blind, placebo-controlled clinical trial designed to evaluate A4250 in 60 patients, ages 6 months to 18 years, with PFIC (subtype 1 or 2), elevated serum bile acid (sBA) levels and pruritus, and an open-label extension study to assess long-term safety and durability of response. Patients in the double-blind trial will receive a 40 or 120 μg/kg oral dose of A4250 or placebo once daily for 24 weeks. The primary endpoint for the US Food and Drug Administration (FDA) evaluation will be an assessment of change in pruritus, and the primary endpoint for the European Medicines Agency (EMA) evaluation will be sBA responder rate.

The team at Albireo is proud to start a Phase 3 trial with A4250 in PFIC to generate data to support potential approval and provide the first approved treatment for patients with PFIC

Ron Cooper, President and Chief Executive Officer of Albireo

“The team at Albireo is proud to start a Phase 3 trial with A4250 in PFIC to generate data to support potential approval and provide the first approved treatment for patients with PFIC,” said Ron Cooper, President and Chief Executive Officer of Albireo. “Our team is committed to working expeditiously to activate clinical trial sites around the world, complete the study and seek regulatory approval as soon as possible.”

A4250 has received orphan drug designation for PFIC in the United States and European Union, and has been granted access to the EMA’s PRIority MEdicines (PRIME) program for the treatment of PFIC.

About the A4250 Phase 3 PFIC ProgramAlbireo's Phase 3 PFIC program includes a single randomised, double-blind, placebo-controlled, multicentre clinical trial and an open-label long-term extension study. The double-blind trial, called PEDFIC-1, is designed to enrol approximately 60 patients with PFIC (subtypes 1 or 2), ages 6 months to 18 years, at sites in the United States, Canada, Europe, the Middle East and Australia. Patients will be assigned to receive either 40 µg/kg/day or 120 µg/kg/day of A4250 or placebo, once daily for 24 weeks. The trial has a primary endpoint for US purposes, a different primary endpoint for EU purposes and several secondary endpoints. The primary endpoint for FDA evaluation, and a key secondary endpoint for EMA evaluation, is an assessment of change in pruritus using a proprietary tool developed by Albireo. The trial's primary endpoint for EMA evaluation, and a key secondary endpoint for FDA evaluation, is sBA responder rate. Patients in the trial will have the opportunity to participate in the PEDFIC-2 open-label extension study to assess long-term safety and durability of response.

About A4250 A4250 is a first-in-class product candidate being developed to treat rare paediatric cholestatic liver diseases and is in Phase 3 development in its initial target indication, progressive familial intrahepatic cholestasis (PFIC). A highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), A4250 has minimal systemic exposure and acts locally in the gut. A4250 has been granted orphan drug designation for PFIC in the United States and European Union. The European Medicines Agency (EMA) has also granted A4250 access to the PRIority MEdicines (PRIME) program for the treatment of PFIC, and its Paediatric Committee has agreed to Albireo's A4250 Paediatric Investigation Plan. A4250 is currently being evaluated in a Phase 3 clinical program in patients with PFIC (subtype 1 or 2).